Ongoing Clinical Trials for Efzofitimod
ATYR1923-C-004: EFFICACY AND SAFETY OF INTRAVENOUS EFZOFITIMOD IN PATIENTS WITH PULMONARY SARCOIDOSIS
We are currently conducting a Phase 3 clinical trial called EFZO-FIT™ that is investigating the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis, which is a major form of interstitial lung disease (ILD).
Pulmonary sarcoidosis is an inflammatory disease of unknown cause, characterized by the formation of granulomas, clumps of inflammatory cells, in the lungs. If left untreated, this inflammation can lead to scarring (fibrosis) of the lung tissue resulting in permanent lung damage and in some cases death. Efzofitimod works by dampening immune responses in inflammatory disease states, with the goal of resolving chronic inflammation and preventing progression of fibrosis. Efzofitimod’s mechanism of action targets the cellular pathology central to pulmonary sarcoidosis.
The EFZO-FIT™ study is a global Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of intravenous (IV) efzofitimod 3 mg/kg and 5 mg/kg versus placebo in patients with symptomatic pulmonary sarcoidosis. This is a 52-week study with patients receiving either efzofitimod or placebo once a month for a total of 12 doses. The study will enroll adults with histologically confirmed pulmonary sarcoidosis receiving stable treatment with oral corticosteroid, with or without immunosuppressant therapy.
The study intends to enroll 264 patients at centers in the United States, Europe, Japan and Brazil. The study will incorporate a forced steroid taper. The purpose of this study is to evaluate whether efzofitimod treatment can reduce patients’ dose of oral corticosteroid compared to placebo. The study will also evaluate if efzofitimod will improve lung function and symptoms compared to placebo when steroids are reduced.
ATYR1923-C-005: STUDY TO EVALUATE THE EFFICACY, SAFETY AND TOLERABILITY OF EFZOFITIMOD IN PATIENTS WITH SYSTEMIC SCLEROSIS (SSC)-RELATED ILD (SSC-ILD)
We are currently conducting a Phase 2 clinical trial called EFZO-CONNECT™ that is investigating the efficacy, safety and tolerability of efzofitimod in patients with systemic sclerosis (SSc, or scleroderma)-related ILD (SSc-ILD).
SSc is a chronic, progressive autoimmune disease characterized by inflammation and fibrosis of connective tissues throughout the body, including the skin and other internal organs. SSc that occurs in the lungs is a form of ILD called SSc-ILD. ILD is the primary cause of death in patients with SSc. Efzofitimod may help resolve inflammation and prevent the progression of fibrosis in patients with SSc-ILD. In an animal model of SSc, efzofitimod significantly reduced lung and skin fibrosis, and efzofitimod has also been shown to inhibit cytokines central to the immune pathology of SSc-ILD, including interleukin-6 (IL-6), in both animal and humans.
This is Phase 2, multicenter, randomized, double-blind, placebo-controlled proof-of-concept study of intravenous (IV) efzofitimod 270 mg and 420 mg versus placebo in patients with SSc-ILD. This is a 28-week study with patients receiving either efzofitimod or placebo once a month for a total of 6 doses. The study will enroll adults with SSc and ILD with progressive disease on background mycophenolate therapy.
The study intends to enroll 25 patients at centers throughout the United States. The purpose of this study is to evaluate the efficacy of multiple IV doses of efzofitimod on pulmonary, cutaneous and systemic manifestations in patients with SSc-ILD. The study will also assess the safety and tolerability of multiple doses of IV efzofitimod in patients with SSc-ILD.
For more information, please visit clinicaltrials.gov (NCT05892614).
Completed Clinical Trials for Efzofitimod (ATYR1923)
ATYR1923-C-002: Investigational Trial of ATYR1923 for the Treatment of Pulmonary Sarcoidosis
In September 2021, we announced positive results from a Phase 1b/2a clinical trial evaluating efzofitimod (ATYR1923) in patients with pulmonary sarcoidosis.
The Phase 1b/2a trial was a multiple-ascending dose, double-blind, placebo-controlled study evaluating efzofitimod in 37 patients with pulmonary sarcoidosis. The purpose of this study was to determine if efzofitimod was safe and tolerable in patients with pulmonary sarcoidosis who underwent a protocol-guided oral corticosteroid taper. This study was also designed to explore whether efzofitimod had the potential to reduce oral corticosteroid use, as well as the effects efzofitimod may have had on pulmonary sarcoidosis by measures of lung function, health-related quality of life assessments and disease biomarkers.
Efzofitimod was safe and well-tolerated in this study and demonstrated dose dependent improvements compared to placebo on multiple efficacy endpoints, including measures of steroid burden, lung function and quality of life. Data from this study were published in the peer-reviewed journal CHEST in October 2022.
aTyr collaborated with the Foundation for Sarcoidosis Research (FSR), the nation’s leading nonprofit organization dedicated to finding a cure for sarcoidosis and improving care for sarcoidosis patients.
ATYR1923-C-003: Investigational Trial of ATYR1923 for the Treatment of COVID-19 Related Severe Respiratory Complications
In the first quarter of 2021 we announced positive data from our Phase 2 clinical trial evaluating efzofitimod in patients with COVID-19 related severe respiratory complications. The trial was a randomized, double-blind, placebo-controlled study designed to investigate the safety and preliminary efficacy of efzofitimod as compared to placebo in 32 hospitalized COVID-19 patients with severe respiratory complications who did not require mechanical ventilation. Patients enrolled in the trial were randomized 1:1:1 to receive a single intravenous dose of either 1.0 or 3.0 mg/kg efzofitimod or placebo. Patients were followed for 60 days post treatment.
ACTRN12617001446358: EVALUATION OF THE SAFETY, TOLERABILITY, IMMUNOGENICITY, PHARMACOKINETICS AND PHARMACODYNAMICS OF INTRAVENOUS ATYR1923 IN HEALTHY VOLUNTEERS
In the second quarter of 2018 we announced positive data from our Phase 1 clinical trial of efzofitimod. This first-in-human, randomized, double-blind, placebo-controlled study was designed to investigate the safety, tolerability, immunogenicity and pharmacokinetics of intravenous efzofitimod in healthy volunteers. The Phase 1 study enrolled 36 healthy volunteers who were randomized to one of six cohorts and received a single infusion of intravenous efzofitimod or placebo. Doses of efzofitimod ranged from 0.03 mg/kg up to 5.0 mg/kg. The results indicate that the drug was generally well-tolerated at all dose levels tested, with no significant adverse events or induction of anti-drug antibodies observed following efzofitimod dosing or throughout the one-month follow-up period.
The pharmacokinetics (PK) of efzofitimod following single-dose administration were linear across the evaluated dose range. Higher efzofitimod doses yielded sustained serum concentrations through the end of the one-month follow-up period that were above the predicted therapeutic threshold, supporting the potential for a once-monthly dosing regimen.
EVALUATION OF THE SAFETY, PHARMACOKINETICS AND IMMUNOGENICITY OF KRP-R120 IN HEALTHY JAPANESE VOLUNTEERS
Kyorin Pharmaceutical, our partner for development and commercialization of efzofitimod for ILD in Japan, conducted a Phase 1 clinical trial of efzofitimod, which investigated the safety, PK and immunogenicity of efzofitimod (known as KRP-R120 in Japan) in 32 healthy Japanese volunteers. In this study, efzofitimod was observed to be generally safe and well-tolerated with no drug-related serious adverse events and PK findings were consistent with previous studies of efzofitimod.